Clinical Trial Updates (SCD)

Gene Therapy

Gene Therapy

Gene Editing

Gene Editing

L-glutamine

L-glutamine

Update: 30 June 2021 The Saudi Food and Drug Authority (SFDA), in Saudi Arabia, will review the oral medication Endari(L-glutamine) for treating sickle cell disease (SCD). Given that the SFDA’s review process…
Xromi

Xromi

  Update: 30 June 2021 No update available   Update: 08 January 2021 No update available   Update: 05 November 2020 No update available   Update: 25 August 2020 No…
Mitapivat

Mitapivat

Update: 30 June 2021 Agios is initiating its pivotal Phase 2/3 study with mitapivat in sickle cell disease by year-end 2021. Source: https://www.globenewswire.com/news-release/2021/06/11/2245660/31990/en/Agios-Announces-Updated-Data-from-ACTIVATE-and-ACTIVATE-T-Phase-3-Studies-of-Mitapivat-in-Pyruvate-Kinase-PK-Deficiency-at-the-European-Hematology-Association-Virtual-Congress.html   Update: 08 January 2021 FDA Orphan…
Imara (IMR-687)

Imara (IMR-687)

  Update: 30 June 2021 Data presented at EHA2021 from the phase 2a clinical trial for the safety and efficacy of IMR-687 in 93 SCD patients showed that: IMR-687 was well-tolerated…
Imatinib

Imatinib

  Update: 30 June 2021 No update available.   Update: 08 January 2021 No update available   Update: 05 November 2020 Imatinib has been shown to drastically increase HbF as…
FT-4202

FT-4202

  Update: 30 June 2021 Data from the presented at EHA2021 showed that 88% of patients (7/8)  have achieved a hemoglobin increase of more than 1 g/dL, with a mean…
FTX-6058

FTX-6058

Update: 30 June 2021 Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total haemoglobin. Fulcrum has initiated a Phase 1 trial with FTX-6058…
Adakveo (Crizanlizumab)

Adakveo (Crizanlizumab)

Update: 30 June 2021 No update available   Update: 08 January 2021 Data presented at ASH2020 confirm that no new safety signals were identified in the enrolled SCD population treated…
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